They Rallied Around ‘Our Boys’ as They Pushed for a Duschenne Cure. Where Did That Leave the Girls?

August 26, 2019

(STAT News) – For generations, a Duchenne diagnosis was a final verdict. There were no meaningful treatments. But now multiple companies are developing gene therapies that, if they are shown to be effective, could help patients make a form of the protein they’re lacking, fundamentally altering their lives. For maybe the first time ever, there is genuine excitement in the Duchenne field. Families of girls affected by the disease, however, fear the advances might pass them by.